Article

Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells

2023-09-02

Abstract excerpt

Alpha-thalassemia is an autosomal recessive disease with increasing worldwide prevalence. The molecular basis is due to mutation or deletion of one or more duplicated α-globin genes, and disease severity is directly related to the number of allelic copies compromised. The most severe form, α-thalassemia major (αTM), results from loss of all four copies of α-globin and has historically resulted in fatality in uter...

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Literature Corpus work: 1010e891-feb1-5398-99f3-83f43b227386
DOI: 10.1101/2023.09.01.555926

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